Publicaciones en las que colabora con Ana Sampedro Pascual (4)
2020
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Brain ventricular enlargement in human and murine acute intermittent porphyria
Human molecular genetics, Vol. 29, Núm. 19, pp. 3211-3223
2013
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Safety and liver transduction efficacy of raav5-cohpbgd in nonhuman primates: A potential therapy for acute intermittent porphyria
Human Gene Therapy, Vol. 24, Núm. 12, pp. 1007-1017
2011
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Sustained enzymatic correction by rAAV-mediated liver gene therapy protects against induced motor neuropathy in acute porphyria mice
Molecular Therapy, Vol. 19, Núm. 2, pp. 243-250
2010
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Porphobilinogen deaminase over-expression in hepatocytes, but not in erythrocytes, prevents accumulation of toxic porphyrin precursors in a mouse model of acute intermittent porphyria
Journal of Hepatology, Vol. 52, Núm. 3, pp. 417-424